Our Discoveries

Live-cell fluorescence imaging showing Rabin8, labeled green, binding to Rab11a, labeled red, minutes after serum starvation in

Akt protein kinase pathway regulates key step in the initiation of cilia formation

CCR investigators have discovered that activating the Akt protein kinase pathway stabilizes the binding of the WDR44 protein to the Rab11 protein. This prevents Rab11 from binding to the Rabin8 protein, thereby blocking cilia formation. When Akt is inactive, though, Rab11 instead is bound by FIP3, enhancing its binding to Rabin8, which helps initiate cilia formation. Since abnormalities in cilia formation are associated with a number of types of cancer, these findings point to several potential targets for cancer therapy.

Bacterial compound targets cancer-promoting RNA

Screening more than 3,000 natural products identified a compound that stops the growth of cancer cells by targeting a growth-promoting microRNA.

Photomicrograph showing graft-versus-host disease in the skin

Cyclophosphamide works in an unexpected way to curb graft-vs-host disease in mice

For years it has been assumed that cyclophosphamide helps reduce the severity of graft-versus-host disease by eliminating alloreactive T cells, but new evidence reveals a different reason.

Majority of patients with advanced multiple myeloma respond to CAR T-cell therapy targeting an anti-B-cell maturation antigen in a phase I trial

A phase I clinical trial of 33 patients infused with a CAR T-cell therapy using T cells genetically engineered to express an anti-B-cell maturation antigen (BCMA) CAR showed that 85 percent of patients had the burden of their advanced multiple myeloma cut by half or more.

Heritable mutations may indicate responsiveness to platinum chemotherapy in malignant mesothelioma

In a genetic analysis of 434 patients with malignant mesothelioma who had undergone platinum chemotherapy, those who carried heritable mutations in BAP1and other DNA repair genes had significantly longer overall survival than those without these mutations. The findings hint at personalized approaches to the disease.

Three-dimensional structure of HIV infected and uninfected T cells interacting

Envelope glycoprotein mutations allow HIV-1 to escape antiretroviral therapy in a lab-based study

CCR investigators have discovered that HIV-1 can compensate for a variety of mutations that block its replication by acquiring mutations in the envelope glycoprotein. Because antiretroviral therapies work by inhibiting viral replication, these findings may have implications for HIV-1 drug resistance.

MEK inhibitor selumetinib granted breakthrough designation by FDA to treat neurofibromatosis type 1 in pediatric patients

The U.S. Food and Drug Administration last week granted breakthrough therapy designation for the MEK 1/2 inhibitor selumetinib. The designation is for the treatment of pediatric patients aged three years and older with neurofibromatosis type 1 (NF1) symptomatic and/or progressive, inoperable plexiform neurofibromas (PN), a rare, incurable genetic condition.

Artistic rendering of a T cell immersed in the tumor microenvironment exposed to high levels of potassium

Harnessing T-cell “stemness” could enhance cancer immunotherapy

A new study led by Nicholas Restifo, M.D., Senior Investigator in CCR’s Surgery Branch, sheds light on one way tumors may continue to grow despite the presence of cancer-killing immune cells. The findings, published March 29, 2019, in Science, suggest a way to enhance the effectiveness of immunotherapies for cancer treatment.

Samples from acute myeloid leukemia patients with the splicing factor mutation

CCR researchers discover how protein translation disruptions can lead to cancer

A mutation in the gene U2AF1, which codes for a protein involved in cutting out key pieces of RNA, can also have widespread effects on the translation of messenger RNA into proteins. A close look at one affected protein, interleukin 8 (IL8), revealed increased production as a result of the mutation, leading to inflammation and cancer spread.

Drug found effective in mice for uveal melanoma

Drugs that directly target the transcription factor YAP, which is associated with the growth of uveal melanoma, are too toxic to be used in patients. A new study in mice, however, finds a new, safe and effective way for targeting the YAP pathway.

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